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Lookup NU author(s): Dr Vicky Brocklebank, Dr Sally Johnson, Dr Patrick Walsh, Dr Edwin Wong, Dr Larissa Kerecuk, Professor Tim Goodship, Professor Neil SheerinORCiD, Professor Kevin MarchbankORCiD, Professor David KavanaghORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Factor H autoantibodies can impair complement regulation, resulting in atypical hemolytic uremic syndrome, predominantly in childhood. There are no trials investigating treatment, and clinical practice is only informed by retrospective cohort analysis. Here we examined175 children presenting with atypical hemolytic uremic syndrome in the United Kingdom and Ireland for factor H autoantibodies which included 17 children with titers above the international standard. Of the 17, seven had a concomitant rare genetic variant in a gene encoding a complement pathway component or regulator. Two children received supportive treatment; both developed established renal failure. Plasma exchange was associated with a poor rate of renal recovery in seven of 11 treated. Six patients treated with eculizumab recovered renal function. Contrary to global practice, immunosuppressive therapy to prevent relapse in plasma exchange treated patients was not adopted due to concerns over treatment associated complications. Without immunosuppression, the relapse rate was high (five of seven). However, reintroduction of treatment resulted in recovery of renal function. All patients treated with eculizumab achieved sustained remission. Five patients received renal transplants without specific factor H autoantibody targeted treatment with recurrence in one who also had a functionally significant CFI mutation. Thus, our current practice is to initiate eculizumab therapy for treatment of factor H autoantibody mediated atypical hemolytic uremic syndrome rather than plasma exchange with or without immunosuppression. Based on this retrospective analysis we see no suggestion of inferior treatment, albeit the strength of our conclusions is limited by the small sample size.
Author(s): Brocklebank V, Johnson S, Sheerin TP, Marks SD, Gilbert RD, Tyerman K, Kinoshita M, Awan A, Kaur A, Webb N, Hegde S, Finlay E, Fitzpatrick M, Walsh P, Wong EKS, Booth C, Kerecuk L, Salama A, Almond M, Inward C, Goodship TH, Sheerin N, Marchbank KJ, Kavanagh D
Publication type: Article
Publication status: Published
Journal: Kidney International
Year: 2017
Volume: 92
Issue: 5
Pages: Pages 1261-1271
Print publication date: 01/11/2017
Online publication date: 24/07/2017
Acceptance date: 28/04/2017
Date deposited: 10/04/2017
ISSN (print): 0085-2538
ISSN (electronic): 1523-1755
Publisher: Elsevier Inc.
URL: https://doi.org/10.1016/j.kint.2017.04.028
DOI: 10.1016/j.kint.2017.04.028
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